Paul Knoepfler, Ph.D.

 Paul  Knoepfler, Ph.D.


  • Associate Professor
  • Cell Biology and Human Anatomy

Stem and Cancer Cell Epigenetics

Research Summary

The general focus of our lab's research is on  the question "What controls stem and cancer cell behavior?" We are interested in answering this question about stem cells both during normal embryonic development but also during healing and regeneration. Further, we are analyzing how this control system goes awry during diseases such as developmental disorders and cancer. Although our lab is mostly focused on studying neural stem cells we are also beginning to examine other types such as embryonic stem cells. One important reason for studying a variety of types of stem cells is that it enables us to ask whether the same types of control system are at work in different stem cells. So far we have found that a particular group of proteins called the Myc family appear to be master regulators of stem cell function. These Myc proteins are not only of critical importance for the ability of stem cells to do their normal "job" but also when the Myc genes are present at too high a concentration in stem cells they cause many human cancers. However we don't have a clear understanding of how Myc genes control the normal function of stem cells in development and regeneration nor how an excess of Myc leads to cancer. Therefore studying Myc's function in stem cells may provide critical clues as to how stem cells can be used in regenerative medicine as well as how to do our best to avoid cancer as a side effect from stem cell based regenerative medicine. Finally, these studies of Myc should also help us to better understand human cancer in a more general sense, hopefully moving us one step closer to a cure.

Select Publications

2010 Knoepfler PS and Bush KM. Software for the efficient

management of mouse Colonies. Dis Model Mech. 3(1-2): 9-10.

2010 Kuwahara A, Hirabayashi Y, Knoepfler PS, Taketo MM, Sakai J,

Kodama T, and Gotoh, Y. Regulation of basal progenitors by Wnt-N-Myc

signaling in the developing neocortex. Development 2010 137(7):1035-44.

2010 Varlakhanova NV, Cotterman RF, deVries WN, Morgan J, Donahue

LR, Murray S, Knowles BB, and Knoepfler PS. myc maintains embryonic stem

cell pluripotency and self-renewal. 2010 Differentiation 80(1): 9-19.

2010 Wey, A. and Knoepfler PS. c-myc and N-myc in brain development.

Aging 2(5): 261-2.

2010 Wey, A. and Knoepfler PS. c-myc and N-myc promote active stem

cell metabolism and cycling as architects of the developing brain. Oncotarget

Jun;1(2):120-30. COVER

2010 Wey, A Ceredeno VM, Pleasure D, and Knoepfler PS. c-myc and

N-myc regulate self-renewal, pluripotency, and metabolism of neural stem cells

to direct brain growth. Cerebellum (4):537-47.

2010 Knoepfler PS. Arrestin’ the hedgehog: Shh limits its own signaling

via B-arrestin1. 2010. Cell Cycle. 9(21):4260-1.

2011 Dominguez-Frutos E., Lopez Hernandez I., Vendrell V., Gallozzi

M., Gutsche K., Sharpe J., Knoepfler PS, Eisenman RN, Trumpp A., and

Schimmang T. N-myc controls proliferation, morphogenesis and patterning of the

inner ear. J. NeuroSci. 2011 31(19):7178-89.

2011 Barrilleaux B. and Knoepfler PS. Overexpression of oncogenes in

human cells using ecotropic lentivirus for enhanced biosafety. J Vis Exp. 2011

Jul 24;(53).

2011 Knoepfler PS. My year as a stem cell blogger. Nature. 2011 Jul


2011 Barrilleaux B. and Knoepfler PS. Inducing iPS cells to escape the

dish. Cell Stem Cell. 2011 Aug 5;9(2):103-11.

2011 Varlakhanova NV, Cotterman RF, Bradnam K, Korf I, and

Knoepfler PS. Myc and Miz-1 have coordinate genomic functions including

targeting Hox genes in human embryonic stem cells. Epigenetics & Chromatin.

2011 Nov 4;4(1):20.


Pshenichnaya ISchouwey KArmaro MLarue LKnoepfler PSEisenman RNTrumpp ADelmas V, and Beermann F. Constitutive gray hair in Mice induced by Melanocyte-specific deletion of c-Myc. 2012 Pigment Cell Melanoma Res. 2012 Mar 15.

Martínez-Cerdeño V, Lemen JM, Chan V, Wey A, Lin W, Dent SR, and Knoepfler PS. N-Myc and GCN5 regulate significantly overlapping transcriptional programs in neural stem cells. PLoS One. 2012;7(6):e39456.

Knoepfler PS. Key anticipated regulatory issues for clinical use of human induced pluripotent stem cells. Regenerative Medicine. 2012. In Press.

Riggs JW,
Barrilleaux B,
Varlakhanova N,
Bush K,
Chan V,
and Knoepfler PS. Induced pluripotency and oncogenic transformation are related processes. 2012. Stem Cells & Dev. In Press


Institute of Pediatric Regenerative Medicine/Shriners Hospital

UC Davis Cancer Center

UC Davis Genome Center

Cell and Developmental Biology